1. 014 AVXS-101 gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): study update. (29th July 2019) Authors: Farrar, Michelle; Swoboda, Kathryn J; Schultz, Meredith; McMillan, Hugh; Parsons, Julie; Alexander, Ian E; Kernbauer, Elaine; Farrow, Marcia; Ogrinc, Francis G; Feltner, Douglas E; McGill, Bryan E; Spector, Sidney A; L'Italien, James; Sproule, Douglas M; Strauss, Kevin A Journal: Journal of neurology, neurosurgery and psychiatry Issue: Volume 90(2019)e7 Page Start: A5 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. 15.33 AVXS-101 in presymptomatic spinal muscular atrophy (SMA). Issue 12 (14th November 2019) Authors: Strauss, Kevin A; Swoboda, Kathryn J; Farrar, Michelle A; McMillan, Hugh J; Parsons, Julie; Krueger, Jena M; Iannaccone, Susan T; Chiriboga, Claudia A; Kwon, Jennifer M; Saito, Kayoko; Scoto, Mariacristina; Kausar, Imran; Schultz, Meredith; Kernbauer, Elaine; Farrow, Marcia; Ogrinc, Francis G; Ka... Journal: Journal of neurology, neurosurgery and psychiatry Issue: Volume 90:Issue 12(2019) Page Start: e7 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗